Mohamed S. Abdelbaki, MD

The study is now open at 7 U.S. sites, including the University of California, San Franciso (UCSF), Washing University in St. Louis/St. Louis Children’s Hospital, Indiana University/Riley Children’s Hospital, University of Alabama Birmingham, Johns Hopkins Hospital, Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center, and Seattle Children’s Hospital. Three additional U.S. sites, including the Children’s Hospital of Philadelphia (CHOP), the Children’s Hospital of Los Angeles (CHLA), and Nationwide Children’s Hospital, are expected to open soon. Three patients have been enrolled in the trial since March 2025. The first two patients successfully completed the initial 30-day safety period without experiencing any dose-limiting toxicities (DLTs), while the third patient began treatment in early November. The team has completed next generation sequencing of tumor samples from all three patients and will continue to expand these analyses as more samples become available.

To broaden access to the trial, the team has successfully amended the study to include additional brain tumor types, and work is underway to open the study internationally in Israel, Australia, and New Zealand. Following the completion of the third patient’s safety window, the team anticipates advancing to the next dose level in mid-December. Additionally, they plan to expand enrollment to include children ages 1-8, allowing younger patients, particularly those with recurrent medulloblastoma and ATRT, to participate in this promising study.

The study is now open for enrollment at five institutions across the country, including UCSF, Washing University in St. Louis/St. Louis Children’s Hospital, Indiana University/Riley Children’s Hospital, University of Alabama Birmingham, and Johns Hopkins Hospital. The first patient was enrolled in March 2025 and has safely completed the first cycle of treatment-receiving three NK cell infusions with no serious side effects. To expand access and accelerate enrollment, the team is actively working to open additional sites nationally and internationally, with several expected to launch in the coming months. Additionally, they have made a key change to the study protocol to allow enrolment of patients with a wider range of brain tumors, including infra- tentorial, multifocal, and metastatic disease, ensuring that more patients have the opportunity to benefit from this promising new therapy.

The team expects that the 6 participating institutions who have successfully completed site initiation visits will open the study within the next few weeks and enroll the first patient soon after. These include UCSF, CHOP, CHLA, Washington University in St. Louis, Indiana University, and University of Alabama Birmingham. The team is continuing to work with additional PNOC sites that have expressed interest in participating to launch the trial, including international sites such as Princess Maxima (Netherlands) and IPEN Shaare Zedek (Israel).